Earlier this year, 109 members of the House of Representatives sent a powerful letter to the FDA urging them to “utilize all available tools, resources, and authorities” in reviewing a treatment for Duchenne muscular dystrophy. Congressional champions Congressmen Mike Fitzpatrick (R-PA), Bill Keating (D-MA), and Peter King (R-NY) led this incredible bipartisan letter. It was because of YOU and your advocacy that we were able to get 109 signatures!

In an effort to make the message from Congress bicameral (both House and Senate) with one unified voice, our long-standing champions in the Senate, Senator Roger Wicker (R-MS) and Senator Amy Klobuchar (D-MN) are leading a similar Senate letter urging the FDA to utilize all available resources and authorities to accelerate the process of getting safe and effective treatments to Duchenne patients. Senator Elizabeth Warren (D-MA) has already signed on as an original co-signer!

The clinical and research community have accelerated therapy development to the point of FDA review.

Our families have spent years participating in the natural history studies and clinical trials needed to generate evidence of these products safety and efficacy profiles.

Congress has ensured that regulators have the resources and authorities to review these products through legislation called FDASIA. (Food and Drug Safety and Innovation Act passed in 2012, which included regulatory flexibility tools for rare disease.)